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ASX:ANP | OTC:ATHJY ANNUAL GENERAL MEETING 2019 For personal use only

For personal use only · ASX:ANP | OTC:ATHJY. For personal use only. ANNUAL GENERAL MEETING 2019. 2. This presentation contains forward-looking statements regarding the Company’sbusiness

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ASX:ANP | OTC:ATHJY

ANNUAL GENERAL MEETING 2019

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This presentation contains forward-looking statements regarding the Company’s business & the therapeutic & commercialpotential of its technologies & products in development. Any statement describing the Company’s goals, expectations,intentions or beliefs is a forward-looking statement & should be considered an at-risk statement. Such statements are subjectto certain risks & uncertainties, particularly those risks or uncertainties inherent in the process of developing technology & inthe process of discovering, developing & commercializing drugs that can be proven to be safe & effective for use as humantherapeutics, & in the endeavor of building a business around such products & services. Actual results could differ materiallyfrom those discussed in this presentation. Factors that could cause or contribute to such differences include, but are not limitedto, those discussed in the Antisense Therapeutics Limited Annual Report for the year ended 30 June 2019, which is availablefrom the Company or at www.antisense.com.au.

FORWARD LOOKING STATEMENTS

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ANTISENSE THERAPEUTICS OVERVIEW

Australian, Melbourne-based

biopharmaceutical company developing &

commercialising antisense

pharmaceuticals for large unmet markets

Advanced stage product pipeline with

positive Phase II clinical results

delivered from two compounds (ATL1102

& ATL1103)

Substantial shareholders include renowned

Australian institutions in life sciences Australian Ethical Investment &

Platinum Asset Management &

Australian biotech pioneer Leon Serry

Phase II clinical trial in Duchenne

Muscular Dystrophy (DMD)* – ATL1102

trial at Royal Children’s Hospital

Melbourne, Australia positive preliminary

results reported

Potential for out-licensing of ATL1103 for

acromegaly.

Preliminary interest from pharmaceutical

companies

*DMD is one of the most common fatal genetic disorders caused by a mutation in the muscle dystrophin gene leading to severe progressive muscle loss & premature death in boys – high unmet medical needF

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ANTISENSE – WHAT IS IT & HOW DOES IT WORK?

Antisense oligonucleotide drugs are small (12-25 nucleotides) DNA or RNA-like compounds that are chemically modified to create medicines

Antisense drugs prevent the production of proteins involved in disease processes by interrupting the translation phase of the protein production which results in a therapeutic benefit to patients

ANP is partnered with Ionis Pharmaceuticals (IONS: market capitalisation:US$9 Billion), world leaders in antisense drug development & commercialisationF

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Targeting diseases where there is a need for improved therapiesANTISENSE THERAPEUTICS ADVANCED STAGE CLINICAL PIPELINE

ATL1102 IN DMD• Conducting Phase II

clinical trial at Royal Children’s Hospital in Melbourne, Australia

• Positive preliminary results reported

• Dosing completed in all 9 patients

ATL1103 IN ACROMEGALY• Phase II clinical trial

completed• Potential for out-

licensing to support and fund further clinical development

ATL1102 IN MS• Phase II clinical trial

completed• Monitoring data from

DMD trial to inform on future clinical development in MS

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ATL1102: DRUG, TARGET & ACTIVITY OVERVIEW

20mer phosphorothioate backbone

5’ 3’

2’-o-methoxyethyl ribose“2’-MOE”

2’-o-methoxyethyl ribose“2’-MOE”

2’-deoxy “gap”

3 9 8

RNaseH active

• ATL1102 is designed to inhibit CD49d expression on lymphocytes and thereby reduce their survival, activation and migration from the blood into sites of inflammation

• ATL1102 is an designed to inhibit CD49d expression on lymphocytes and thereby stop/restrict there migration from the blood into sites of inflammation (e.g. the CNS in Multiple Sclerosis patients as pictured below) to reduce or modulate the adverse inflammatory effects

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o Duchenne Muscular Dystrophy (DMD) is a devastating genetic muscular disease caused by loss of dystrophin with progressive muscle wasting & associated muscle injury leading to inflammation &fibrosis (100% mortality)

o Affects boys with an incidence of ~1 in 3,500 & prevalence of ~44,000 in US & EU

o Dystrophin restoration treatments recently approved – eteplirsen (Exondys 51:Sarepta Therapeutics) for the 13% of patients amenable to Exon 51 skipping

o Key challenge in management of DMD patients is to reduce the inflammation that exacerbates muscle fibre damage

o Corticosteroids (CS) are the only therapy used to treat the inflammation in DMD but have insufficient efficacy& significant side effects including weight gain, reduced bone density & growth retardation. CS not as effective in patients with a greater number of CD49d receptors on T cells.

Source: CureDuchenne

WHAT IS DMD?F

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WHY ATL1102 for DMD?

ATL1102, an antisense drug to CD49d, shown to be a highly active immunomodulatory drug with potent effects on inflammatory processes in MS patients

• 90% reduction in inflammatory brain lesions vs placebo[Limmroth V et al Neurology 2014]

• Reduced CD49d on T & B cells, and T & B cell numbers by ~25 & 50% respectively

• Pre-clinical & clinical data in MS has supported move directly into the six-month DMD patient trial (effective leveraging of substantial investment & progress made to date in MS)

Pivotal scientific publication confirming CD49d as a potential target for DMD therapy

• DMD patients with greater number of circulating T cells with high levels of CD49d (alpha chain of VLA-4) expression have both more severe & rapid progression of disease [Pinto-Mariz et al Skeletal Muscle 2015]

• Ambulant patients on CS suggesting CS do not reduce CD49dhi expression on T cells

• CS treatment does not modulate CD49d expression on T cells in MS

• Non-ambulant DMD patients have greatest number of CD49d high expressing T cells

• Improved therapies are needed to ameliorate DMD severity & delay disease progression• DMD is an orphan indication so can benefit from IP & development incentives

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ATL1102 PHASE II STUDY

Study Title:A Phase 2 open label study to determine the safety, efficacy and pharmacokinetic profile of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne Muscular Dystrophy. (ACTRN12618000970246)

Primary objective: To assess the safety and tolerability of 25 mg of ATL1102 administered once weekly (s.c. injection) for 24 weeks in non-ambulatory DMD participants

Secondary objectives: To evaluate the• lymphocyte-modulatory potential of ATL1102 in participants with DMD• PK profile of ATL1102 in participants with DMD• effects of ATL1102 on functional capacity in participants with DMD• effects of ATL1102 on respiratory function in participants with DMD• effects of ATL1102 on quality-of-life in participants with DMD

Design:

Single-centre, open-labelled study conducted at the Royal Children’s Hospital (RCH), Melbourne, Australia

Sample size:

9 participants

Target population:• participants diagnosed with DMD and have been non-ambulatory for at least 3 months• 10 to 18 years of age• body weight of more than 25 kg and less than or equal to 65 kg

• Trial being led by RCH Head of Neuromuscular Clinic Professor Monique Ryan & RCH Neuromuscular Fellow Dr Ian Woodcock • Neuromuscular clinic at RCH the largest in the Southern Hemisphere for treating boys with DMD

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ATL1102 PHASE II STUDY - SAFETY OVERVIEWPreliminary Data Presented by Dr Ian Woodcock at Action Duchenne Conference Nov 2019 on first 7 boys to have completed dosing

An Independent Data and Safety Monitoring Board (DSMB) provides safety oversight for the study

• The DSMB consists of 3 paediatricians and a biostatistician, the DSMB is chaired by A/Professor Andrew Kornberg, MD

• The DSMB evaluate safety data on an ongoing basis and based upon the evaluation, issue formal recommendations for continuation/modification/discontinuation of the study to the sponsor

• Based on their review of the data to date, the DSMB have no safety concerns

ATL1102 appears to be generally well tolerated

• No Serious Adverse Events (SAEs) have been reported

• No participants have been withdrawn from the study for safety related reasons

• The most commonly reported TEAEs were injection site erythema (8 participants), skin discolouration (7 participants), injection site pain (5 participants) and injection site bruising (4 participants)F

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ATL1102 PHASE II STUDY Overview of Efficacy Parameters - Preliminary Data

The Secondary Objectives of the study include evaluation of the following efficacy parameters:• Lymphocyte-modulation potential to be determined by assessing number and percentages of

lymphocytes, CD4+ and CD8+ T cells and, CD4+ CD49d and CD8+ CD49d T cells • Early indications of an immunomodulatory effect have been observed where T-cell numbers (in

particular those expressing the CD49d, the biological target of ATL1102) are trending downward during the 24-week treatment phase while returning to around starting levels post dosing

• Effects of ATL1102 on functional capacity in participants with DMD • Evaluation of muscle function via Performance of Upper Limb Test (PUL 2.0)• Evaluation of muscle strength via MyoGrip and MyoPinch assessments (using the Myoset System)

- data presented on the following slide

• Effects of ATL1102 on respiratory function in participants with DMD• Includes % predicted Peak Expiratory Flow (PEF) and % predicted Forced Expiratory Volume (FVC)

- data presented on the following slideFor

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ATL1102 PHASE II STUDY Overview of Efficacy Parameters - Preliminary Data

Change from Baseline to Week 24

Patient No. PUL 2.0 MyoGrip

(dom) (Kg)

MyoGrip(dom)

(% Pred)

MyoPinch(dom)(Kg)

MyoPinch(dom)

(% Pred)

% Predicted FVC

% Predicted PEF

01-001 +2 -0.63 -4.49 0.03 -0.62 -3.20 6.3001-002 +2 0.22 0.49 -0.02 -0.29 -14.8 -17.301-003 0 0.68 1.02 -0.40 -6.59 -9.10 8.7001-004 +2 1.09 1.01 0.37 2.99 0.80 7.2001-006 -3 -0.27 -0.60 0.07 0.94 -6.50 6.9001-008 +7 1.00 1.11 0.30 2.77 -7.70 -18.210-009 0 -0.33 -3.75 -0.22 -4.97 -9.10 -4.3001-01001-011

Mean Change(95% CI):

1.4(-1.39, 4.25)

0.3(-0.38, 0.89)

-0.7(-2.95, 1.47)

0.0(-0.23, 0.27)

-0.8(-4.23, 2.58)

-7.09(-11.6,-2.53)

-1.53(-12.5, 9.46)F

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ATL1102 Phase II StudyEfficacy Parameters – Comparison to Published Data for Myoset Assessments

* Ricotti et. al 2016 . PLoS One, 11(9) e0162542 historical results from 8 Non – Ambulant patients on CS for 6month

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ATL1102 PHASE II STUDY Summary of Safety and Efficacy Parameters - Preliminary Data

• ATL1102 appears to be generally safe and well tolerated in non-ambulant boys with DMD

• No Serious Adverse Events (SAEs) have been reported

• The DSMB, responsible for safety oversight of the study, have no safety concerns

• To date the most commonly reported TEAEs were injection site erythema (8 participants), skin discolouration (7 participants), injection site pain (5 participants) and injection site bruising (4 participants)

• ATL1102, a novel antisense drug being developed for the treatment of inflammation that exacerbates muscle fibre damage in DMD, appears to be demonstrating positive effects on disease progression parameters

• Data from the first 7 participants completing dosing shows an apparent improvement in muscle strength based on the change from baseline after 24 weeks of dosing with ATL1102 as assessed by MyoGrip and MyoPinch compared to the loss of muscle strength reported in the literature in similar patient populations

• The preliminary data is also suggestive of an improvement in muscle function as assessed by the Performance of Upper Limb Test (PUL 2.0), where the majority of participants have demonstrated either improvements or no change in their PUL 2.0 scores from baseline after 24 weeks of dosing with ATL1102

• Promising preliminary results support continued development of ATL1102 for the treatment of DMD

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ATL1102 PHASE II STUDY Reporting of data from all 9 subjects post completion of dosing

• All 9 participants have completed their 6 months of dosing

• The trial is continuing with patients in the 2 month monitoring phase or follow up period of the trial

• The Last Participant Last Visit (in follow up period) for the study will be beginning of Jan 2020

• Results from the completion of dosing in all patients remain on track to be reported before the end of the year

• Data is to be reviewed by the Board during their AGM associated visit and expected to be reported soon thereafter

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PHASE IIB CLINICAL TRIAL

• Scientific Advice (SA) meetings held with three European regulatory authorities

• SA meetings focussed on the Phase IIb trial design, dose escalation plans, applicability of the study end-points and the study duration. ANP expects to receive written responses month following each meeting

• General acceptance by the agencies on the trial efficacy endpoints (Pul2, Myoset), safety monitoring plan, dosing duration (1 year) and the use of higher doses

• Clarification provided by the agencies that the above could be a path forward to an approval on positive Phase IIb results

• Next step is to follow up development plan with the European Medicines Agency (EMA) and subsequent to the finalisation of the results from the current Phase II trial, engage with the Food and Drug Administration (FDA) on development plans for the USF

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• Prospect for these therapies to be complementary rather than competitive• Other anti-inflammatory therapies are being tested in ambulant children

TREATMENT DEVELOPMENT FOCUSING ACROSS ALL INTERVENTION POINTS

Sarepta Therapeutics (Nasdaq:SRPT), Wave Life Sciences (Nasdaq:WVE)

ANTI-FIBROTICSINFLAMMATION & FIBROSIS

CARDIAC & CALCIUM REGULATION

MUSCLE GROWTH & REGENERATION

STEROIDS

ATL1102

CAP-1002

MYOSTATINMIGF1

NOVEL & EXISTING CARDIAC DRUGS

IDEBENONE

GENE THERAPY

ETEPLIRSEN EXON 51 SKIPPINGATALUREN READTHROUGH THERAPY

DYSTROPHIN RESTORATION & REPLACEMENT

DMDPfizer, Sarepta, Solid Biosciences (Nasdaq:SLDB)

PTC Therapeutics (Nasdaq:PTCT)

Santhera (SWX:SANN)

Pfizer (Nasdaq:PFE)

Capricor Inc (Nasdaq:CAPR)

INTERVENTION POINTS TREATMENT PROVIDERS

RESPIRATORY PROBLEMS

Sarepta Therapeutics (Nasdaq:SRPT)Wave Life Sciences (Nasdaq:WVE)

ATL1102’s novel mechanism in targeting CD49d suggests potential for drug to be used in combination with other treatments including anti-inflammatory agents

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• ATL1102 - anti-inflammatory and immune modulating agent with potential for multiple clinical applications

MARKET CONSIDERATIONS FOR ATL1102

ANTI-INFLAMATORYAnti-Inflammatory Therapeutics Market^ is expected to garner US$106.1 billion by 2020(Allied Market Research)^MS, Arthritis, Psoriasis, Respiratory, IBD

CORTICOSTEROIDSThe global steroid market is forecast to attain the value of US$17 Billion by the end of 2025 (QV Research)

DMD THERAPIESThe global DMD drug market is expected to reach over US$4 Billionby 2023(Grand View Research)

• Corticosteroids are the only marketed therapy to treat the inflammatory damage associated with dystrophin loss in DMD

• Prevalence of DMD in EU and US est. 44,000 with most ambulant and ~2/3 of non-ambulant patients on corticosteroids*

• DMD cost of therapy considerations

Deflazacort (Emflaza) is a CS approved in US only - average annual cost estimated > US$80K per patient per annum

Exondys 51 (dystrophin restoration agent) cost in the US is > US$400K per patient per annum

* Cowan L et al BMC Neurology (2019), 1-10

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• Cost per patient of Exondys 51 is US$300K/year

• 2nd quarter 2019 total net revenue for Exondys 51 –US$94.7 million

• Mr William Goolsbee, ex Chairman of Sarepta, is a non-executive director of Antisense Therapeutics

VALUE CREATION POTENTIAL OF ATL1102 FOR DMD

EXONDYS 51 (DEVELOPED BY SAREPTA) APPROVED BY THE FDA IN LATE 2016 UNDER THE

ACCELERATED APPROVAL PATHWAY

• Approval based on the surrogate endpoint of dystrophin increase in skeletal muscle observed in some Exondys 51-treated patients

• Sarepta market capitalisation has grown from ~US$60m (July 2012) to $3 billion on FDA approval of Exondys 51-today ~US$6.5 billion

• Exondys 51 – despite being first FDA approved treatment for DMD is only useful in 13% of boys with the exon 51mutation

• Inflammation (the target of ATL1102 in DMD) contributes to disease progression in all DMD patients

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DMD & INDUSTRY CONFERENCESMeetings ANP may present at and or attend include:

DMD related scientific conferences• SOS ACTT Duchenne Conference in Melbourne, Australia, March 2020 • Muscular Dystrophy Association (MDA) conference in Orlando, Florida US, March 2020• American Academy of Neurology (AAN) in Toronto, Canada, April-May 2020 • PPMD conference, Arizona, US, June 2020• World Muscle Society Congress Halifax, Canada, Sept/October 2020 • Action Duchenne Conference November in the United Kingdom (2020 tbd)• TREAT-NMD Conference in Leiden, Netherlands (2020 tbd)

Industry investor/partnering meetings• Bio Partnering J.P. Morgan annual conference in San Francisco, US, 13-16 January 2020• Europe Spring conference in Paris, France, March 2020• Bio Asia-Tokyo, Japan, March 2020• Bio International Convention conference San Diego, US, June 2020 • Bio-Europe Conference in Munich, Germany, October 2020

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ATL1102 for OTHER AUTOIMMUNE – INFLAMMATORY DISEASES

• CD49d is clinically validated in Multiple Sclerosis and Crohns disease where antibody drugs to CD49d are used

• There are several orphan indications (like DMD) where CD49d expression is important in disease processes, and where CD49d appears to be a superior target

• ANP could move directly into clinical studies based on existing preclinical and clinical data

• Grant funding opportunities exist for such projects

• ATL1102 drug product is available for studies

• ANP to progress in indications where there are ATL1102 platform (antisense) and target (CD49d) based advantages

• Further details to be advised once appropriate Intellectual Property protection is in place

• ATL1102 targets CD49d+ immune cells involved in disease processes • CD49d is a clinically validated target in multiple disease indications and a potential superior

target in other autoimmune inflammatory disease• Pipeline development focus

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ANPOB LISTED OPTIONS & CAPITAL MANAGEMENT UPDATE

• ANPOB options monies received to date (>$650K) plus commitments to exercise from holders including Key Management Personnel will take total to in excess of $1million

• Further option exercise anticipated ahead of option expiry on 19 December 2019

• R&D tax credit of $559K received

• Based on above, ANP will be funded into 2H’20 calendar year including costs associated with European regulatory interactions to prepare the application for the Phase IIb DMD trial

• Additionally a non-discounted funding facility has been Board approved to provide supplementary capital at Company’s discretion

• Stock to be issued within available placement capacity

• At a price and volume set by the Company

• No obligation on ANP to utilize

• To be accessed if and when required

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BOARD OF DIRECTORS

Mr Robert W MosesIndependent Non-Executive Chairman

Formerly Corporate Vice President of CSL Limited. Mr. Moses draws on more than 40 years’ experience in the pharmaceutical/biotechnology industry.

Mr Mark Diamond Managing Director & Chief Executive Officer

Over 30 years’ experience in the pharmaceutical & biotechnology industry. Formerly Director, Project Planning/Business Development at Faulding Pharmaceuticals in the USA, Senior Bus Dev Manager within Faulding's European operation & International Business Development Manager with Faulding in Australia.

Dr Graham Mitchell Independent Non-Executive Chairman

Joint Chief Scientist for the Victorian Government Department of Environment & Primary Industries. Formerly Director of Research in the R&D Division of CSL Limited.

Dr Gary Pace Independent Non-Executive Director

Dr Pace has more than 40 years’ international experience in the development & commercialisation in biotechnology/pharmaceuticals industries. Long-term board level experience with both multi-billion & small cap companies.

Mr William Goolsbee Independent Non-Executive Director

Founder, Chairman & CEO of Horizon Medical Inc. 1987 –2002 until acquisition by UBS Private Equity. Founding Director then Chairman of ImmunoTherapy Corporation until acquisition by AVI Biopharma, Inc. (now Sarepta Therapeutics). Former Chairman of privately held BMG Pharma LLC & Metrodora Therapeutics.

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KEY FINANCIALS

Market Capitalisation (at $0.08) A$34M

Shares on issue 423.6M

52-week high/low $0.145 - $0.023

Options (ANPOB, $0.08 exp. 19/12/19) 64.3M

Cash as at 30 September 2019 $2M

OWNERSHIP STRUCTURETop 40 holders 54.27%

Substantial Shareholders

• Australian Ethical Investment 16.04%

• Platinum Asset Management 6.27%

• Leon Serry 6.15%

CORPORATE OVERVIEWF

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ANTISENSE THERAPEUTICS SUMMARY & VALUE DRIVERS

Phase II clinical trial in Duchenne Muscular Dystrophy (DMD) – ATL1102

− Trial is fully enrolled and all patients have completed dosing

− Positive preliminary results reported from first 7 patients having completed dosing

− Phase IIb trial design and approval process running in parallel to potentially accelerate development of ATL1102

− Drug potentially complementary to other DMD programs e.g. Sarepta Therapeutics

− Significantly ‘underserved market’ with comparable company benchmarks demonstrating substantial value creation potential

Advanced stage product pipeline – two compounds with positive Phase II clinical results published in high quality peer reviewed scientific journals with multiple clinical applications

Highly regarded Australian institutional shareholders - Australian Ethical Investment & Platinum Asset Management

ATL1103 (atesidorsen) for acromegaly − Potential for partnering to further develop the compound

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For more information:Mark DiamondManaging Director+61 (0) 3 9827 8999www.antisense.com.au

Investment enquiriesGennadi KoutchinXEC Partners+61 423 500 [email protected]

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